Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Everyone’s favorite little blue pill may offer more than just a boost in the bedroom. New research suggests the erectile ...

A presidential directive issued in December 2025 has triggered a sweeping revision of the U.S. childhood immunization ...

Children and adolescents diagnosed with high blood pressure have double the risk of major cardiovascular problems as adults, a new study that followed them into their 20s and 30s reports. It’s another ...

A revolutionary treatment brings new hope to those suffering from a devastating rare skin disorder, while showcasing an innovative funding model that could transform rare disease research In a ...

Leigh syndrome is a rare metabolic disorder that begins in childhood and causes severe neurological and muscular problems. The disease has long been considered untreatable. In a pilot study involving ...

People can develop celiac disease at any age. Symptoms in children may vary depending on how old they are. Symptoms are most likely to begin once an infant starts to eat gluten. Celiac disease can ...

Arthritis is often associated with older age, but it also affects children. One of the most common forms is juvenile idiopathic arthritis (JIA), an inflammatory condition that causes persistent joint ...

Australia's Murdoch Children's Research Institute is helping scientists use stem cell medicine and artificial intelligence to develop precision therapies for pediatric heart disease, the leading cause ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Imagine waiting years to find out what is wrong with your child. For families dealing with rare disease, this is a harsh reality, and the journey is often long and filled with uncertainty. On average, ...

Please provide your email address to receive an email when new articles are posted on . Black children and those of more than one racial background were less likely to achieve LLDAS and had higher ...